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Rare Pediatric Disease Priority Review Voucher
Pharma
Sentynl, Cyprium score FDA nod for 1st med for Menkes disease
The FDA has approved Sentynl and Cyprium's Zycubo as the first therapy in the U.S. for the rare genetic neurodegenerative disorder Menkes disease.
Kevin Dunleavy
Jan 13, 2026 10:45am
House vote moves FDA's rare PRV program a step closer to revival
Dec 2, 2025 11:22am
Bavarian Nordic's $160M PRV price reflects new realities
Jun 18, 2025 11:47am
Zevra lines up $150M from sale of PRV tied to Miplyffa approval
Feb 27, 2025 9:58am
Bluebird has pawned off the second of two PRVs for $95M
Jan 6, 2023 10:19am
